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Scientists treated three adults and six children with absent or severe reduced mutations in a Phase I/II trial with an intrabone administration of hematopoietic stem cells transduced with the lentiviral vector GLOBE. [Nat Med]
Abstract

Researchers developed EDIT-101, a candidate genome-editing therapeutic, to remove the aberrant splice donor created by the IVS26 mutation in the CEP290 gene and restore normal CEP290 expression. Key to this therapeutic, they identified a pair of Staphylococcus aureus Cas9 guide RNAs that were highly active and specific to the human CEP290 target sequence. [Nat Med]
Abstract

To assess efficacy in vivo, the authors used adeno-associated virus-mediated expression of the most potent SARM1 dominant-negative and nerve transection as a model of severe axonal degeneration. [J Exp Med]
Abstract | Press Release

Investigators demonstrated that heparan sulfate mimetic injections that restore the extracellular matrix network and enhance the biological activity of growth factors, associated with local injection of mesenchymal stromal cell (MSC) protected in a hydrogel, that increased cell engraftment and cell survival, improved the therapeutic benefit of MSC treatment in two animal models relevant of the human pathology. [Sci Rep]
Full Article

The authors give an overview of the most recent developments in the field of T-cell genetic engineering including TCR-gene transfer and CAR T-cells strategies. [Adv Drug Deliv Rev]
Abstract | Graphical Abstract

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Marker Therapeutics, Inc. reported that data from four abstracts—including three oral presentations—were accepted for presentation. [Press release from Marker Therapeutics, Inc. discussing research presented at the Transplantation & Cellular Therapy (TCT) Meetings of the American Society for Blood and Marrow Transplantation and the Center for International Blood and Marrow Transplant Research (ASBMT and CIBMTR), Houston]
Press Release

Sernova Corp. announced the enrollment of the first three of seven subjects in its Phase I/II trial of Sernova’s Cell Pouch™ for Clinical Islet Transplantation in type 1 diabetes. [Sernova Corp.]
Press Release

AVROBIO, Inc. announced that its investigational gene therapy candidate for cystinosis, AVR-RD-04, is cleared to begin a Phase I/II clinical trial, following acceptance of the IND application by the FDA. [AVROBIO, Inc.]
Press Release

Thyas Co. Ltd. signed a licensing agreement with iPS Academia Japan, Inc. Under the agreement, Thyas obtains exclusive rights on patents related to production and use of iPS cell-derived T cells, and non-exclusive rights on the patents related to iPS cell generation, for development and commercialization of autologous immunotherapy of cancers and infectious diseases. [Thyas Co. Ltd. (GlobeNewswire, Inc.)]
Press Release

The FDA plans to hire at least 50 new clinical reviewers tasked with assessing cell and gene therapies to prepare for what the agency describes as a surge of cutting-edge products currently entering early development. [FierceBiotech]
Press Release

Many U.S. government scientists and federally funded researchers breathed a sigh of relief last month, after the partial shutdown of the U.S. government began. That’s because the budget impasse between Congress and President Donald Trump didn’t affect some of the largest federal research agencies, including the $39 billion National Institutes of Health (NIH) and the $35.6 billion Department of Energy (DOE). [Science Insider]
Editorial

British scientists are ramping up preparations for a ‘no deal’ Brexit on 29 March — a situation that would instantly affect travel, data collection, clinical trials and crucial laboratory supplies. [Nature News]
Editorial

FDA

Statement from FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research on New Policies to Advance Development of Safe and Effective Cell and Gene Therapies (FR Doc. No:PressAnnouncements) Notice